An examination of systems of access to important high cost medicines: a critical analysis of the nationally subsidised scheme of access to tumour necrosis factor inhibitors in Australia

Background: Access to 'high-cost medicines' under Australia's Pharmaceutical Benefits Scheme (PBS) is characterised by strict eligibility criteria. The PBS access scheme for the anti-rheumatic biologicals (etanercept, infliximab, and adalimumab) was examined for concordance with Australia's National Medicines Policy.Methods: Semi-structured interviews with a range of stakeholders were conducted. National, aggregated prescription and expenditure data from Medicare Australia and dispensing data from the Drug Utilisation Sub-Committee were analysed. Access to biologicals was also examined from an ethical perspective.Results: Interviewees agreed that controlled access to high-cost medicines was broadly equitable and practical but specific concerns included: timeliness of access; bureaucracy of the process; contentious cases of individual patients being denied access; insufficient patient information; the quantum of resources required to administer the access scheme; inadequate stakeholder consultation. The access requirement of a history of failure of conventional anti-rheumatic drugs was supported. Recommendations included proactive review of the access criteria and outcomes; greater transparency and formal stakeholder involvement to increase public confidence in the definition of 'target patient population' and a formal appeal mechanism to increase the fairness and accountability of the PBS. Establishment of an appeal mechanism is supported by 'accountability for reasonableness' framework grounded in procedural justice.Data needed to examine the health outcomes associated with the use of biologicals on a national level was not easily available. This shortcoming is discordant with National Medicines Policy. Utilisation of biologicals over the first two years of PBS-subsidy was conservative but with considerable variability across States and Territories (an 8-fold difference between the jurisdictions), usage roughly correlating with access to rheumatologists. Introduction of PBS-subsidised biologicals did not alter the trends in utilisation of non-biological anti-rheumatic drugs.Conclusions: This research suggests that policy-makers focus upon: explicitly considering ethical principles and formally involving stakeholders when developing policies on access to high-cost medicines; improving communication and providing information based on increased transparency; and establishing formal mechanisms for review of and appeals against PBS decisions. The comprehensive evaluation of medicine use and outcomes post-subsidy is critical for the future of the PBS. The National Medicines Policy has proved a useful framework for evaluating this access scheme